Blueprint Medicines potentially delays Ayvakit decision; Control beats treatment in mesothelioma trial
→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering. Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III VOYAGER before they give a judgment.
→ Targovax has released early data on their experimental drug for mesothelioma, and they’re not great. The trial tested ONCOS-102 on 31 patients, including one cohort of first-line patients and one cohort of second-line patients. The response rate was higher in the control groups for both first-line and second-line patients, than in the two treatment groups. Targovax said those numbers may have been skewed by the sample size, and they saw promising clinical activity – a common refrain from drug companies faced with a failed trial, but one that may have some merit here: There were only 5 second-line patients in the control group and they had an eye-popping 60% response rate from the standard-of-care (as opposed to 11% in the ONCOS-102 group). Targovax said they will now explore combining their adenovirus-based therapy with a checkpoint therapy.
→ Jazz Pharmaceuticals has submitted an NDA for their narcolepsy drug, JZP-258, and redeemed a priority review voucher for it. The drug is designed to treat daytime sleepiness and cataplexy – sudden muscle weakness, often in response to a trigger – in patients over the age of 7. The point of the drug is to bring the same efficacy as Jazz’s long-running narcolepsy treatment Xyrem but with 1,000 to 1,500 milligrams less sodium. Xyrem, or sodium oxybate, generally pushes patients’ sodium intake well over the American Heart Association’s recommended 1,500 milligrams per day, potentially causing long-term cardiovascular effects.